In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease

Park, Hanseul; Oh, Jungju; Shim, Gayong; Cho, Byounggook; Chang, Yujung; Kim, Siyoung; Baek, Soonbong; Kim, Hongwon; Shin, Jeain; Choi, Hwan; Yoo, Junsang; Kim, Junyeop; Jun, Won; Lee, Minhyung; Lengner, Christopher J.; Oh, Yu-Kyoung; Kim, Jongpil

doi:10.1038/s41593-019-0352-0

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In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease

Park, Hanseul;
Oh, Jungju;
Shim, Gayong;
Cho, Byounggook;
Chang, Yujung;
... Kim, Jongpil;
외 11명

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초록

In vivo gene editing in post-mitotic neurons of the adult brain may be a useful strategy for treating neurological diseases. Here, we develop CRISPR-Cas9 nanocomplexes and show they were effective in the adult mouse brain, with minimal off-target effects. Using this system to target Bacel suppressed amyloid beta (A beta)-associated pathologies and cognitive deficits in two mouse models of Alzheimer's disease. These results broaden the potential application of CRISPR-Cas9 systems to neurodegenerative diseases.

키워드

MICE; EXPRESSION; PROTEIN; MUSCLE

제목: In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease

저자: Park, Hanseul; Oh, Jungju; Shim, Gayong; Cho, Byounggook; Chang, Yujung; Kim, Siyoung; Baek, Soonbong; Kim, Hongwon; Shin, Jeain; Choi, Hwan; Yoo, Junsang; Kim, Junyeop; Jun, Won; Lee, Minhyung; Lengner, Christopher J.; Oh, Yu-Kyoung; Kim, Jongpil

DOI: 10.1038/s41593-019-0352-0

발행일: 2019-04

유형: Article

저널명: Nature Neuroscience

권: 22

호: 4

페이지: 524 ~ 528